BioPharm Brief: Gene Therapy Progress, FDA Approval, and $103M Funding

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Today, we’re looking at new gene therapy data, an at-home treatment option for thyroid eye disease, and a major funding boost for kidney disease research.

First, Opus Genetics reported sustained vision improvements with its investigational gene therapy OPGx-LCA5 in an early-stage study of patients with an inherited retinal disease that can cause severe vision loss. Improvements in visual function were observed through 24 months in adults, supporting plans to advance the therapy into Phase 3 at the lowest dose tested.

Next, the FDA has approved Roche’s Enspryng, a subcutaneous monoclonal antibody, as the first at-home treatment option for adults with thyroid eye disease. The approval gives patients a way to receive treatment by injection at home after training, offering an alternative to infusion-based care. The decision was supported by two Phase 3 trials, although only one met its primary endpoint.

And finally, Estuary Biotherapeutics has launched with $103 million in financing to develop gene therapies for autosomal dominant polycystic kidney disease, or ADPKD. Its two programs target the underlying genetic causes of the disease, with EST-201 expected to enter clinical trials in 2027 and EST-101 in 2028. The approach aims to restore key proteins in kidney cells rather than simply slow disease progression.

That’s today’s BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways

• Gene therapy advances: Opus Genetics reported sustained vision improvements through 24 months with OPGx-LCA5, supporting plans for a Phase 3 trial.
• At-home treatment option: The FDA approved Roche’s Enspryng as the first at-home subcutaneous treatment for thyroid eye disease.
• Major funding for kidney disease: Estuary Biotherapeutics launched with $103 million to advance gene therapies for autosomal dominant polycystic kidney disease.